New CAR T case study shows promise in acute myeloid leukemia

TAMPA, Fla. – Chimeric Antigen Receptor T-cell therapy, also known as CAR T therapy, was named the biggest research breakthrough of 2017 by the American Society of Clinical Oncology. The personal gene therapy utilizes a patient's own immune cells to fight cancer. The Food and Drug Administration has approved CAR T therapy products for adults with diffuse large B-cell lymphoma and pediatric and young adults suffering from acute lymphoblastic leukemia. Now, researchers at Moffitt Cancer Center are working to expand this revolutionary therapy to other cancers.

One possible disease that could benefit is acute myeloid leukemia (AML), the most common acute leukemia affecting adults. More than half of AML patients go into remission after chemotherapy. However, many relapse because residual leukemia cells can evade chemotherapy and the immune system. A new phase 1 trial called the THINK (THerapeutic Immunotherapy with NKG2D) study is investigating Celyad's new CAR T therapy CYAD-01 which genetically modifies immune cell to express a natural killer receptor that targets leukemia tumors.

According to a case study from trial published online ahead of print in the journal Haematologica, a patient has remained cancer free for nine months after being treated with CYAD-01, followed by a bone marrow transplant.

"It is important to note this is the first time CAR T has induced a remission in AML," said case study author David Sallman, M.D., assistant member of the Department of Malignant Hematology at Moffitt. "It is also the first time CAR T has been effective without the need of preconditioning chemotherapy."

For CAR T treatment, T cells are removed from a patient's blood and sent to a lab where the cells are genetically modified to better enable them to identify and attack cancer cells. Once the new cells are received, patients typically receive preconditioning chemotherapy to deplete their immune system to make room for the new cells. In the THINK study, no preconditioning chemotherapy was necessary.

"Our case study shows that CAR T therapy is a viable option for AML patients," added Sallman. "We now need to take what we have learned in this initial study and expand the trial."

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The THINK study is sponsored by Celyad.

Moffitt is dedicated to one lifesaving mission: to contribute to the prevention and cure of cancer. The Tampa-based facility is one of only 49 National Cancer Institute-designated Comprehensive Cancer Centers, a distinction that recognizes Moffitt's scientific excellence, multidisciplinary research, and robust training and education. Moffitt is a Top 10 cancer hospital and has been nationally ranked by U.S. News & World Report since 1999. Moffitt devotes more than 2 million square feet to research and patient care. Moffitt's expert nursing staff is recognized by the American Nurses Credentialing Center with Magnet® status, its highest distinction. With more than 5,700 team members, Moffitt has an economic impact in the state of $2.1 billion. For more information, call 1-888-MOFFITT (1-888-663-3488), visit MOFFITT.org, and follow the momentum on Facebook, Twitter and YouTube.